Induced Pluripotent Stem Cells for Neuromuscular Diseases: Potential for Disease Modeling, Drug Screening, and Regenerative Medicine

Neuromuscular diseases are a group of acquired or inherited diseases affecting the peripheral nervous system and the skeletal muscle. The large majority of these diseases are currently incurable. Neuromuscular disease modeling is quite challenging due to the lack of appropriate human disease models. Induced pluripotent stem cell technology has not only enabled the potential to recapitulate human disease in the Petri dish, thus allowing for more specific drug screening, but also opened the opportunity for the development of autologous cell-based therapy. In this article, we will highlight major research advances and future prospects in these areas.